No More Waiting: MEPs insist on action at Rare Disease Day event
5 March 2025, Brussels – Today, policymakers, patient advocates, and healthcare leaders gathered at the European Parliament to mark Rare Disease Day 2025, calling for urgent EU action to address the unmet needs of the 30 million people living with a rare disease in Europe.
Co-hosted by MEPs Stine Bosse (Renew, Denmark) and Adam Jarubas (EPP, Poland) in collaboration with EURORDIS-Rare Diseases Europe, the event – ‘Impact of Rare Diseases: More Than You Can Imagine’ – highlighted the need for stronger EU policies, funding, and cooperation to tackle diagnostic delays, treatment gaps, and social inequalities affecting the rare disease community.
The event opened with a powerful intervention from Adéla Odrihocká of Rare Diseases Czech Republic, who lives with Ehlers-Danlos syndrome and shared her personal experience of a nearly two-decade-long diagnostic odyssey. She highlighted the systemic barriers that people with rare diseases face, from misdiagnosis and delays in accessing care to the absence of social support. “I consider myself lucky to be standing here today because for some in my community, this long diagnostic odyssey was fatal,” she stated.
Alongside these lived experiences, Tim Wilsdon, Vice President at Charles River Associates, presented findings from new research quantifying the economic burden of rare diseases in the EU, estimating the annual cost at €250 billion – six times higher than for non-rare diseases. His analysis reinforced the urgent need for investment in faster diagnosis, better access to treatment, and improved quality of life for people living with rare diseases.
MEPs call for action
Opening the event, MEP Stine Bosse (Renew, Denmark) stressed the European Parliament’s responsibility to take concrete action on rare diseases, warning that inaction comes at a greater cost than investment.
“It has to come together. We have to talk about that elephant in the room – what does it cost? And how do we actually make sure that we get people to understand that it is much more expensive not to do anything than to do things together?” she urged.
She highlighted the importance of cross-border collaboration, emphasising that rare disease patients and their families do not care where a treatment is available – only that it is accessible.
Bosse also stressed the need to involve all stakeholders, including industry as part of the solution, ensuring that medicines reach the people who need them.
“Industry is part of making sure that we earn money, that we have workplaces, that we can compete, and that we have access for our citizens to medicines. That is at the core of what we’re trying to achieve right now.”
Finally, she reaffirmed her commitment to ensuring that rare diseases remain a political priority, even amid broader economic and geopolitical challenges.
“Now, as a full-fledged policymaker, this is a guiding principle for me: that we actually, even in the dire straits that we are finding ourselves in right now in Europe, we must never forget the most vulnerable of the vulnerable.”
“Rare diseases is where working together becomes visibly logical! We need to think European to solve many of the issues we will face”.
In addition, MEP Vlad Voiculescu (Renew, Romania) called for a more coordinated EU approach, stressing that rare disease patients are often marginalised in both health and social policy.
“This is what Europe is about – solidarity. Solidarity between generations, between Member States, between people who have means and people who don’t, between those who are healthy and those who are sick.”
Following key figures relayed from our Rare Barometer survey on newborn screening, he urged policymakers to invest in newborn screening, diagnostic technologies, and stronger cooperation between health systems.
During the discussion on holistic care, MEP Tilly Metz (Greens, Luxembourg) underscored the urgent need to integrate mental health into rare disease policies, stating:
“It’s not only about making people stay alive, but also that they feel alive and feel part of society.”
Metz urged policymakers to ensure better recognition and harmonisation across Member States:
“A big demand towards the post-2025 Disability Strategy is to improve the recognition of disability assessments across Europe, but also to increase the quality and coverage of the assessment in general. We need this harmonisation.”
A European Action Plan for Rare Diseases
Avril Daly, President of EURORDIS, highlighted the need to engage with the new European Commission to ensure rare diseases remain a political priority. She stressed the importance of efficiency and cross-sector collaboration:
“We can do things cheaper and smarter; we can use joined-up thinking – and that’s why it’s so important to have people in the room who can work together and find solutions.”
She also underscored the resilience of the rare disease community:
“Nothing scares us, and as long as we’re together and working together, that is going to get us where we want to go.”
Reflecting the event’s discussions, Virginie Bros-Facer, Chief Executive Officer of EURORDIS, reinforced the need for urgent and coordinated EU action.
“This event showed strong commitment, but discussion alone is not enough – we need a European Action Plan for Rare Diseases with clear objectives, measurable goals, and lasting political commitment. At a time of global instability, rare diseases must remain a political priority.”
She added: “EURORDIS will continue working with the European Parliament Intergroup on Cancer & Rare Diseases to ensure rare disease policy remains at the heart of the EU agenda.”
About EURORDIS-Rare Diseases Europe
EURORDIS-Rare Diseases Europe is a unique, non-profit alliance of over 1,000 rare disease organisations from 74 countries that work together to improve the lives of the 30 million people living with a rare disease in Europe. By connecting people, families, and rare disease groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies, and services.
Contact
Julien Poulain
Communications Manager
EURORDIS-Rare Diseases Europe
Julien.poulain@eurordis.org
+33 6 42 98 14 32